Trial Outcomes & Findings for Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases (NCT NCT01963143)
NCT ID: NCT01963143
Last Updated: 2017-03-29
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
48 participants
Primary outcome timeframe
After a minimum 5 infusions on each product, at pre-infusion, 10 minutes before end of infusion, 1, 3, 6, 24, 48 hours, 4, 7, 14, 21 and 28 days post-infusion
Results posted on
2017-03-29
Participant Flow
Participant milestones
| Measure |
Treatment Sequence 1 - Adults
Gammaplex 5% \& Gammaplex 10 on a 21-day treatment schedule
|
Treatment Sequence 2 - Adults
Gammaplex 10 and Gammaplex 5% on a 28-day treatment schedule
|
Pediatrics
Gammaplex 10 on a 21 or 28 day treatment schedule
|
|---|---|---|---|
|
Overall Study
STARTED
|
14
|
19
|
15
|
|
Overall Study
COMPLETED
|
14
|
18
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases
Baseline characteristics by cohort
| Measure |
Treatment Sequence 1 - Adults
n=14 Participants
Gammaplex 5% and Gammaplex 10 on a 21-day schedule
|
Treatment Sequence 2 - Adults
n=19 Participants
Gammaplex 5% and Gammaplex 10 on a 28-day schedule
|
Pediatrics
n=15 Participants
Gammaplex 10 on a 21 or 28 day schedule
|
Total
n=48 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
17 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
31 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
28 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
20 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
13 Participants
n=206 Participants
|
45 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
48 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
Hungary
|
0 participants
n=99 Participants
|
0 participants
n=107 Participants
|
1 participants
n=206 Participants
|
1 participants
n=7 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=99 Participants
|
19 participants
n=107 Participants
|
14 participants
n=206 Participants
|
44 participants
n=7 Participants
|
|
Region of Enrollment
United Kingdom
|
3 participants
n=99 Participants
|
0 participants
n=107 Participants
|
0 participants
n=206 Participants
|
3 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: After a minimum 5 infusions on each product, at pre-infusion, 10 minutes before end of infusion, 1, 3, 6, 24, 48 hours, 4, 7, 14, 21 and 28 days post-infusionPopulation: PK population
Outcome measures
| Measure |
Gammaplex 10%
n=16 Participants
|
Gammaplex 5%
n=16 Participants
|
|---|---|---|
|
Primary Bioequivalence Analysis - Area Under the Curve Within a 28-day Dosing Interval (AUC0-28) in Adult Subjects
AUC(0-t) absolute
|
1.01 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.98 to 1.03
|
1.01 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.98 to 1.03
|
|
Primary Bioequivalence Analysis - Area Under the Curve Within a 28-day Dosing Interval (AUC0-28) in Adult Subjects
AUC(0-t) baseline-adjusted
|
1.07 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.93 to 1.23
|
1.07 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.93 to 1.23
|
SECONDARY outcome
Timeframe: After a minimum 5 infusions on each product, at pre-infusion, 10 minutes before end of infusion, 1, 3, 6, 24, 48 hours, 4, 7, 14 and 21 days post-infusionOutcome measures
| Measure |
Gammaplex 10%
n=14 Participants
|
Gammaplex 5%
n=14 Participants
|
|---|---|---|
|
Secondary Bioequivalence Analysis - Area Under the Curve Within a 21-Day Dosing Interval (AUC0-21) in Adult Subjects
AUC(0-t) absolute
|
0.99 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.95 to 1.02
|
0.99 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.95 to 1.02
|
|
Secondary Bioequivalence Analysis - Area Under the Curve Within a 21-Day Dosing Interval (AUC0-21) in Adult Subjects
AUC(0-t) baseline-adjusted
|
1.10 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.96 to 1.26
|
1.10 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.96 to 1.26
|
SECONDARY outcome
Timeframe: After a minimum 5 infusions on each product, at pre-infusion.Outcome measures
| Measure |
Gammaplex 10%
n=30 Participants
|
Gammaplex 5%
n=30 Participants
|
|---|---|---|
|
Secondary Bioequivalence Analysis - IgG Trough Levels
28-day dosing interval
|
0.98 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.94 to 1.02
|
0.98 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.94 to 1.02
|
|
Secondary Bioequivalence Analysis - IgG Trough Levels
21-day dosing interval
|
0.95 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.92 to 0.99
|
0.95 ratio Gammaplex 10%/Gammaplex 5%
Interval 0.92 to 0.99
|
Adverse Events
Gammaplex 5% - All Subjects
Serious events: 2 serious events
Other events: 23 other events
Deaths: 0 deaths
Gammaplex 10% - All Subjects
Serious events: 1 serious events
Other events: 44 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Gammaplex 5% - All Subjects
n=33 participants at risk
Subjects aged 17-55 years
|
Gammaplex 10% - All Subjects
n=47 participants at risk
Subjects aged 2-55 years
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neuroendocrine tumour
|
3.0%
1/33 • Number of events 1
|
0.00%
0/47
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
3.0%
1/33 • Number of events 1
|
0.00%
0/47
|
|
Nervous system disorders
IIIrd nerve paralysis
|
3.0%
1/33 • Number of events 1
|
0.00%
0/47
|
|
Immune system disorders
Anaphylactic reaction
|
0.00%
0/33
|
2.1%
1/47 • Number of events 2
|
Other adverse events
| Measure |
Gammaplex 5% - All Subjects
n=33 participants at risk
Subjects aged 17-55 years
|
Gammaplex 10% - All Subjects
n=47 participants at risk
Subjects aged 2-55 years
|
|---|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
6.1%
2/33 • Number of events 2
|
17.0%
8/47 • Number of events 8
|
|
Infections and infestations
Nasopharyngitis
|
3.0%
1/33 • Number of events 2
|
10.6%
5/47 • Number of events 7
|
|
Infections and infestations
Viral upper respiratory tract infection
|
12.1%
4/33 • Number of events 5
|
8.5%
4/47 • Number of events 5
|
|
Infections and infestations
Acute sinusitis
|
15.2%
5/33 • Number of events 6
|
6.4%
3/47 • Number of events 3
|
|
Infections and infestations
Chronic sinusitis
|
6.1%
2/33 • Number of events 2
|
8.5%
4/47 • Number of events 4
|
|
Infections and infestations
Sinusitis
|
3.0%
1/33 • Number of events 1
|
14.9%
7/47 • Number of events 7
|
|
Infections and infestations
Influenza
|
0.00%
0/33
|
12.8%
6/47 • Number of events 6
|
|
Infections and infestations
Bronchitis
|
9.1%
3/33 • Number of events 3
|
0.00%
0/47
|
|
Nervous system disorders
Headache
|
18.2%
6/33 • Number of events 16
|
23.4%
11/47 • Number of events 28
|
|
Nervous system disorders
Migraine
|
9.1%
3/33 • Number of events 4
|
6.4%
3/47 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.0%
1/33 • Number of events 2
|
6.4%
3/47 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.1%
2/33 • Number of events 2
|
8.5%
4/47 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
9.1%
3/33 • Number of events 3
|
4.3%
2/47 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
6.1%
2/33 • Number of events 2
|
0.00%
0/47
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/33
|
10.6%
5/47 • Number of events 6
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
9.1%
3/33 • Number of events 3
|
2.1%
1/47 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhoea
|
6.1%
2/33 • Number of events 2
|
4.3%
2/47 • Number of events 2
|
|
Gastrointestinal disorders
Nausea
|
9.1%
3/33 • Number of events 3
|
2.1%
1/47 • Number of events 1
|
|
General disorders
Fatigue
|
9.1%
3/33 • Number of events 7
|
2.1%
1/47 • Number of events 5
|
|
General disorders
Pyrexia
|
0.00%
0/33
|
6.4%
3/47 • Number of events 3
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
6.1%
2/33 • Number of events 2
|
0.00%
0/47
|
|
Psychiatric disorders
Insomnia
|
9.1%
3/33 • Number of events 3
|
0.00%
0/47
|
Additional Information
Head of Medical Information
Bio Products Laboratory
Phone: +44 (0)20 8957 2200
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60